CSL Behring lero yalengeza kuti European Medicines Agency (EMA) yavomereza Marketing Authorization Application (MAA) ya etranacogene dezaparvovec (EtranaDez) pansi pa njira yake yowunikira. Etranacogene dezaparvovec ndi kafukufuku wa adeno-associated virus five (AAV5)-based gene therapy yomwe imaperekedwa ngati chithandizo chanthawi imodzi kwa odwala haemophilia B omwe ali ndi vuto lotaya magazi kwambiri. Ngati avomerezedwa, etranacogene dezaparvovec idzapatsa anthu okhala ndi hemophilia B ku European Union (EU) ndi European Economic Area (EEA) njira yoyamba yochizira jini yomwe imachepetsa kwambiri kuchuluka kwa magazi pachaka pambuyo pa kulowetsedwa kamodzi. Kuwunika kofulumira kungachepetse nthawi yomwe MAA ivomerezedwe kuti iwunikenso ndipo imaperekedwa ngati mankhwala ochiritsira akuyenera kukhala okhudza thanzi la anthu, makamaka zokhudzana ndi luso lachirengedwe.
"Monga woyamba kulandira chithandizo cha jini cha hemophilia B, gawo lofunikira kwambiri pakuwongolera izi likubweretsa CSL Behring kuyandikira gawo limodzi kuti akwaniritse lonjezo la chithandizo chamtundu wa anthu omwe ali ndi vuto la magazi," atero a Emmanuelle Lecomte Brisset, Mtsogoleri wa Global Regulatory Affairs ku CSL Behring. "Tikuyembekezera kugwira ntchito ndi akuluakulu oyang'anira kuti athandize anthu omwe ali ndi vuto losautsali komanso lotha moyo wawo wonse."
MAA imathandizidwa ndi zopeza zabwino kuchokera ku kuyesa kofunikira kwa HOPE-B, kuyesa kwakukulu kwa jini mu hemophilia B mpaka pano. Odwala a Hemophilia B omwe amadziwika kuti ali ndi vuto lotaya magazi kwambiri lomwe limathandizidwa ndi etranacogene dezaparvovec adawonetsa kuchepetsedwa kwa mlingo wamagazi wapachaka (ABR) ndi 64% ndipo adawonetsa kupambana kwamankhwala a prophylaxis pakatha miyezi 18 atalandira chithandizo poyerekeza ndi miyezi isanu ndi umodzi. Kuphatikiza apo, panali kuwonjezeka kokhazikika komanso kokhazikika pamiyezo yantchito ya Factor IX (FIX). Etranacogene dezaparvovec idapangidwa makamaka kuti ipangitse kutsekeka kwa magazi kwanthawi zonse pothana ndi zomwe zimayambitsa vutoli: jini yolakwika ya F6 yomwe imayambitsa kuperewera kwa clotting Factor IX (FIX).
"Kuvomerezedwa kwa etranacogene dezaparvovec kuti iwunikenso ndi EMA kupititsa patsogolo ntchito yathu yosalekeza yopititsa patsogolo miyoyo ndi moyo wa anthu omwe ali ndi hemophilia B ndi matenda ena osowa komanso aakulu," adatero Bill Mezzanotte, Wachiwiri kwa Purezidenti, Mutu wa R&D ndi Chief Medical Officer wa CSL Limited. "Ndife onyadira kugwira ntchito ndi UniQure kukhala patsogolo pa chitukuko cha sayansi chomwe cholinga chake ndi kupanga hemophilia B kukhala gawo lachiwiri la moyo wa wodwala m'malo mokhala ndi nkhawa nthawi zonse."
Chitukuko chachipatala chazaka zambiri chidatsogozedwa ndi uniQure (Nasdaq: QURE) komanso thandizo la mayeso azachipatala ku United States asintha kupita ku CSL Behring atapeza ufulu wapadziko lonse wochita malonda etranacogene dezaparvovec. CSL Behring ili mkati mosinthana ndi thandizo la mayeso azachipatala ku European Union.
ZOMWE MUNGACHITE PA NKHANIYI:
- “We are proud to work with uniQure to be at the forefront of this scientific advancement which aims to make hemophilia B a secondary part of a patient’s life instead of a constant concern.
- If approved, etranacogene dezaparvovec will provide people living with hemophilia B in the European Union (EU) and European Economic Area (EEA) with the first-ever gene therapy treatment option that significantly reduces the rate of annual bleeds after a single infusion.
- Accelerated assessment potentially reduces the timeline once the MAA is accepted for review and is provided for a medicinal product when the therapy is expected to be of major public health interest, particularly pertaining to therapeutic innovation.